Apadamtase alfa is a recombinant human ADAMTS13 for enzyme replacement therapy in patients with congenital thrombotic thrombocytopenic purpura. Thrombotic thrombocytopenic purpura (TTP) is a rare blood clotting disorder characterized by thrombocytopenia, microangiopathic hemolytic anemia, and various degrees of organ damage.
- Mechanism
- Curator reviewed · 3 references
Congenital thrombotic thrombocytopenic purpura (cTTP) can be caused by a severe deficiency of plasma ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13). ADAMTS13 is a plasma zinc metalloprotease that regulates the activity of von Willebrand factor (VWF) by cleaving large and ultra-large VWF multimers to smaller units, reducing the platelet binding properties of VWF and its subsequent formation of microthrombi.
Adzynma (ADAMTS13, recombinant-krhn) is a recombinant form of endogenous ADAMTS13. It is used as an enzyme replacement therapy in patients with cTTP and serves to partially restore ADAMTS13 activity in order to prevent thromboses.
- Primary indication
- Recombinant human ADAMTS13 is indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).Curator reviewed · 1 structured indication
- First approval
- United States, 2023 · European Union, 2025
- Also known as
- ADAMTS13, recombinant-krhn · Metalloproteinase ADAMTS13 (human recombinant BAX930) · rADAMTS-13 · rADAMTS13 · Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifs
- Code names
- BAX-930 · SHP-655 · TAK-755
- Brand names
Resolves to
What you can answer from here — as of June 15, 2026
Which drugs share a target with Apadamtase alfa, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account